Cetuximab Sarotalocan Sodium (Genetical Recombination)

First patient treated in the U.S. Initial enrollment planned for the U.S., Japan, and Taiwan, with further expansion to additional countries and regions SAN DIEGO, Jan. 23, 2025 /PRNewswire/ -- Rakuten Medical, Inc., a global biotechnology company developing and commercializing precision, cell-targeting Alluminox™ platform-based photoimmunotherapy, today announced the initiation of its global multi-regional Phase 3 clinical trial evaluating ASP-1929 photoimmunotherapy in combination with anti-PD-1 (pembrolizumab) as a first-line therapy for recurrent head and neck squamous cell carcinoma (HNSCC) (Protocol number: ASP-1929-381 / Acronym: ECLIPSE* / ClinicalTrials.gov Identifier: NCT06699212). The first patient in this trial recently received a study treatment at the Avera Cancer Institute in Sioux Falls, South Dakota, USA. Alongside the U.S., patient enrollment is slated for sites in Japan and Taiwan, followed by additional countries and regions. The ASP-1929-381 study is a multi-regional multi-center, randomized, open-label Phase 3 trial, designed to assess the efficacy and safety of ASP-1929 photoimmunotherapy in combination with pembrolizumab as a first-line treatment for locoregional recurrent HNSCC without distant metastases. Approximately 400 patients globally will be randomized to either an experimental arm receiving ASP-1929 photoimmunotherapy in combination with pembrolizumab, or a control arm receiving the current pembrolizumab-based standard of care (SOC), where patients may receive pembrolizumab alone or in combination with chemotherapy according to the physician's choice. The primary endpoint is Overall Survival (OS), with key secondary endpoints including Complete Response Rate (CRR) and Overall Response Rate (ORR). This study builds on data from an interim evaluation (data cut-off: August 31, 2023) of 19 patients enrolled in the HNSCC cohort of a prior Phase 1b/2 study (ASP-1929-181 study / ClinicalTrials.gov Identifier: NCT04305795). The data showed an estimated 24-month survival rate of 52.4%, with a median OS not yet reached, and a generally tolerated safety profile. Head and neck cancer ranks as the 7th most common cancer globally with over 90% classified as squamous cell carcinoma1. More than 60% of HNSCC patients present with locally or regionally advanced disease (stage III or IV)1. Of these, 15-40% will experience recurrence, facing a poor prognosis and a 5-year overall survival rate of less than 50%1. Dr. Kristen Coffroth, MD of Avera Medical Group Ear, Nose & Throat - Head and Neck Surgery, and one of the investigators for the ASP-1929-381 study at the Avera Cancer Institute, stated, "Photoimmunotherapy has a unique mode of action, which involves a biophysical disruption of targeted cells. Pembrolizumab has become a mainstay in systemic treatment for recurrent/metastatic HNSCC. This trial will evaluate whether the combination of ASP-1929 photoimmunotherapy with pembrolizumab can harness synergistic antitumor effects, which may shed new light on the treatment of locoregional recurrent HNSCC." Dr. William Chad Spanos, MD, FACS of Avera Medical Group Ear, Nose & Throat - Head and Neck Surgery, and Principal Investigator for the ASP-1929-381 study at the Avera Cancer Institute, added, "I am impressed by the technology behind photoimmunotherapy, which offers a novel and targeted approach to cancer treatment. Combining this with pembrolizumab has the potential to expand first-line treatment options for patients with recurrent HNSCC. I am pleased to initiate this groundbreaking trial at our site and look forward to contributing to advancing care for patients facing this challenging disease." Mickey Mikitani, Chief Executive Officer and Vice Chairman of the Board of Rakuten Medical, concluded, "The initiation of this global Phase 3 trial marks a significant milestone in our journey to contribute to the treatment and lives of patients with head and neck cancer and potentially others affected by cancer. With encouraging data from Phase 1b/2 study, we are optimistic about the potential of ASP-1929 photoimmunotherapy combined with pembrolizumab to improve treatment outcomes in recurrent head and neck cancer." Disclaimer: Rakuten Medical's Alluminox™ platform-based photoimmunotherapy is investigational outside Japan. * ECLIPSE: Eliminating Cancer with Laser Illumination and Photo SEnsitizer Chow LQM. Head and Neck Cancer. N Engl J Med. 2020;382(1):60-72. doi:10.1056/NEJMra1715715 About Rakuten Medical Rakuten Medical, Inc. is a global biotechnology company developing and commercializing precision, cell-targeting Alluminox™ platform-based photoimmunotherapy, which, in pre-clinical studies, has been shown to induce rapid and selective cell killing. Rakuten Medical's photoimmunotherapy is currently investigational outside Japan. Rakuten Medical is committed to its mission to conquer cancer by developing its pioneering treatments as quickly as possible to as many patients as possible all over the world. The company has offices in 5 countries/regions, including the United States, where it is headquartered, Japan, Taiwan, Switzerland and India. For more information, visit . About Alluminox™ platform The Alluminox™ platform is Rakuten Medical's investigational technology platform that combines pharmaceuticals, medical devices, medical technology, and other peripheral technologies. Rakuten Medical is developing Alluminox platform-based photoimmunotherapy, which involves two key steps: 1) drug administration and 2) targeted illumination using medical devices. The drug component consists of a cell-targeting moiety conjugated to a light-activatable dye, such as IRDye® 700DX (IR700), that selectively binds the surface of targeted cells, such as tumor cells. The device component consists of a light source that locally illuminates the targeted cells with red light (690nm) to transiently activate the drug. Rakuten Medical's pre-clinical data have shown that this activation elicits rapid and selective necrosis of targeted cells through a biophysical process that compromises the membrane integrity of the targeted cells. Therapies developed on the Alluminox platform may also result in local and systemic innate and adaptive immune activation due to immunogenic cell death of the targeted tumor cells and/or the removal of targeted immunosuppressive cells within the tumor microenvironment. Photoimmunotherapy was originally developed by Dr. Hisataka Kobayashi and team from the National Cancer Institute in the United States. Outside Japan, Rakuten Medical's Alluminox platform-based photoimmunotherapy is investigational. About ASP-1929 Rakuten Medical's first pipeline drug developed on its Alluminox™ platform is ASP-1929, an antibody-dye conjugate comprised of the antibody cetuximab and IRDye® 700DX, a light activatable dye. ASP-1929 binds to epidermal growth factor receptor (EGFR), a cancer antigen expressed in multiple types of solid tumors, including head and neck, breast, lung, colorectal, prostate and pancreatic cancers. After binding to cancer cells, ASP-1929 is locally activated by illumination with red light (690 nm), emitted by a laser device system to produce a photochemical reaction. This reaction is believed to cause damage to the membrane of cancer cells, leading to selective necrosis of cancer cells. In Japan, ASP-1929 received marketing approval from the Japanese Ministry of Health, Labor, and Welfare for unresectable locally advanced or recurrent head and neck cancer in September 2020, under the Sakigake Designation System and the Conditional Early Approval System. ASP-1929 photoimmunotherapy in combination with pembrolizumab is currently under investigation in a global Phase 3 clinical trial as a first-line therapy for recurrent head and neck cancer. Outside Japan, ASP-1929 has not yet been approved for commercial use by any regulatory authority. Forward Looking Statements This press release contains forward looking statements that correspond to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include various risks, uncertainties, and assumptions that may cause Rakuten Medical's business plans and results to differ from the anticipated results and expectations expressed in these statements. These "forward looking statements" contain information about the status and development of our products, including the Alluminox™ platform, as well as other regulatory and marketing authorization efforts, the potential benefits, efficacy, and safety of therapies created using the Alluminox platform, and the status of regulatory filings. The approval and commercial success of the product may not be achieved. Forward looking statements relate to the potential benefits, efficacy, and safety of our therapies, and the status of regulatory filings. Such statements may include words such as "expect," "believe," "hope," "estimate," "looks as though," "anticipate," "intend," "may," "suggest," "plan," "strategy," "will," and "do", and are based on our current beliefs. In addition, this press release uses terms such as "important," "notable," and "abnormal" to express opinions about clinical trial data. Ongoing clinical trial studies include various risks and uncertainties, in particular, problems that arise during the manufacturing stage of our therapies, the occurrence of adverse safety events, situations in failure to demonstrate therapeutic benefits, and other various risks and uncertainties, both reasonable and unreasonable. For this reason, actual results, including regulatory approvals and uncertainties in the commercialization process of our therapies, may differ from published information. Except to the extent required by applicable law, we undertake no obligation to publicly update this or any other forward-looking statement, whether because of new information, future developments or events, changes in assumptions, changes in the factors affecting forward-looking statements. If one or more forward-looking statement(s) is updated, no inference should be drawn that additional updates will be made to those or other forward-looking statements. Contact Us SOURCE Rakuten Medical, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Mickey Mikitani, as sole CEO, reaffirms his commitment to the company's mission of conquering cancer for patients all over the world Minami Maeda has been appointed as the new President to expedite pipeline development, with a strategic focus on securing FDA approval for ASP-1929, expanding into broader Asia, and further driving commercial growth in Japan A new global Phase 3 clinical trial for ASP-1929 photoimmunotherapy in combination with anti-PD-1 is expected to be initiated in 2H 2024 SAN DIEGO, Aug. 19, 2024 /PRNewswire/ -- Rakuten Medical, Inc., a global biotechnology company developing and commercializing oncology precision, cell-targeting photoimmunotherapy through its proprietary Alluminox™ platform, announces that Hiroshi "Mickey" Mikitani now serves as its sole Chief Executive Officer and Vice Chairman of the Board, and Minami Maeda has been appointed as its new President, effective August 9, 2024. Takashi "Tora" Toraishi has chosen to step down from his role as Co-Chief Executive Officer, President and Board Director. The new management team is set to further advance Rakuten Medical's Alluminox™ platform and prioritize obtaining FDA approval for ASP-1929 as early as possible. The company's new global Phase 3 study of ASP-1929 photoimmunotherapy in combination with anti-PD-1 for first-line recurrent head and neck squamous cell carcinoma (HNSCC) is expected to commence first in the United States in 2H 2024. Rakuten Medical also aims to extend its reach into Asian markets, where many countries face high unmet medical needs, by leveraging its growing expertise from commercial treatments in Japan. Asia accounts for half of the world's cancer incidence and has the highest cancer mortality rate*. Rakuten Medical's strong track record of commercial success in Japan has generated substantial scientific publications, which the company believes would help support new medical communities' better understanding of the Alluminox™ platform. In Japan, the company will continue to drive growth and vigorously support the realization of its strategic agenda in the U.S. and Asia. Mickey Mikitani, Chief Executive Officer and Vice Chairman of the Board of Rakuten Medical, stated, "The appointment of Minami as President and the formation of our new management team underscores our unwavering commitment to advancing our innovation for patients in need. We continue striving to advance cancer treatment through our technology platform, and I am fully committed to further driving the company's vision and strategic direction." "I am honored to take on this pivotal role at such a critical time for Rakuten Medical," said Minami Maeda, President of Rakuten Medical. "Our treatment has positively impacted patients' lives in Japan, as demonstrated by our robust commercial growth there. I am excited to collaborate with our talented team to build an even stronger company and extend our contributions to more patients and broader communities as we continue to pursue our global development efforts." Since joining Rakuten Medical in November 2017, Maeda has demonstrated outstanding leadership and strategic acumen. Most recently serving as Chief Commercial Officer of Rakuten Medical, Inc., and Representative Director & President of Rakuten Medical K.K., he has significantly contributed to commercial growth and established a scalable business model for the company's innovative drug-device combination in Japan. His efforts have greatly enhanced the company's global growth and technological advancements. Prior to his tenure at Rakuten Medical, Maeda gained extensive experience in business and public policy through his work at McKinsey & Company's Tokyo and Johannesburg offices. He also served as Chief of Staff to Japan's Minister of Health, Labor, and Welfare. Rakuten Medical extends its gratitude to Toraishi for his strong leadership and contributions since 2017. His tenure has been marked by significant achievements, including the successful development and the world-first approval of ASP-1929 in Japan, and the establishment of a strong foundation for future growth. Disclaimer: Rakuten Medical's therapies based on Alluminox™ platform are investigational outside of Japan. * Bray F, Laversanne M, Sung H, Ferlay J, Siegel RL, Soerjomataram I, Jemal A. Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin. 2024 May-Jun;74(3):229-263. doi: 10.3322/caac.21834. Epub 2024 Apr 4. PMID: 38572751. About Rakuten Medical, Inc. Rakuten Medical, Inc. is a global biotechnology company developing and commercializing precision, cell targeting therapies based on its proprietary Alluminox™ platform, which, in pre-clinical studies, has been shown to induce rapid and selective cell killing and tumor necrosis. Alluminox therapies have not yet been approved outside of Japan. Rakuten Medical is committed to its mission to conquer cancer by delivering its innovative treatments as quickly as possible to as many patients as possible all over the world. The company has offices in 5 countries/regions, including the United States, where it is headquartered, Japan, Taiwan, Switzerland and India. For more information, visit . About Alluminox™ platform The Alluminox™ platform is an investigational technology platform based on a cancer therapy called photoimmunotherapy, which was developed by Dr. Hisataka Kobayashi and team from the National Cancer Institute in the United States. Rakuten Medical is developing the Alluminox platform as a technology consisting of a drug, device, and other related components. The drug component of the platform consists of a targeting moiety conjugated with one or more dyes leading to selective cell surface binding. The device component consists of a light source that locally illuminates the targeted cells with light to transiently activate the drug. Pre-clinical data have shown that this activation elicits rapid and selective necrosis of targeted cells through a biophysical process that compromises the membrane integrity of the targeted cells. Therapies developed on the Alluminox platform may also result in local and systemic innate and adaptive immune activation due to immunogenic cell death of the targeted cancer cells and/or the removal of targeted immunosuppressive cells within the tumor microenvironment. Outside of Japan, Alluminox therapies have not yet been approved by any regulatory authority. Forward Looking Statements This press release contains forward looking statements that correspond to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements include various risks, uncertainties, and assumptions that may cause Rakuten Medical's business plans and results to differ from the anticipated results and expectations expressed in these statements. These "forward looking statements" contain information about the status and development of our products, including the Alluminox™ platform, as well as other regulatory and marketing authorization efforts, the potential benefits, efficacy, and safety of therapies created using the Alluminox platform, and the status of regulatory filings. The approval and commercial success of the product may not be achieved. Forward looking statements relate to the potential benefits, efficacy, and safety of our therapies, and the status of regulatory filings. Such statements may include words such as "expect," "believe," "hope," "estimate," "looks as though," "anticipate," "intend," "may," "suggest," "plan," "strategy," "will," and "do", and are based on our current beliefs. In addition, this press release uses terms such as "important," "notable," and "abnormal" to express opinions about clinical trial data. Ongoing clinical trial studies include various risks and uncertainties, in particular, problems that arise during the manufacturing stage of our therapies, the occurrence of adverse safety events, situations in failure to demonstrate therapeutic benefits, and other various risks and uncertainties, both reasonable and unreasonable. For this reason, actual results, including regulatory approvals and uncertainties in the commercialization process of our therapies, may differ from published information. Except to the extent required by applicable law, we undertake no obligation to publicly update this or any other forward-looking statement, whether because of new information, future developments or events, changes in assumptions, changes in the factors affecting forward-looking statements. If one or more forward-looking statement(s) is updated, no inference should be drawn that additional updates will be made to those or other forward-looking statements. Contact Us SOURCE Rakuten Medical, Inc.